NEW HOPE RESEARCH FOUNDATION
Providing new hope to those with lysosomal storage diseases.
Programs and results
What we aim to solve
The New Hope Research Foundation was established as a non-profit organization in 2006 to support scientific research and clinical studies needed to help patients neurologically impacted by lysosomal storage diseases. As the initial area of focus, the foundation is conducting and coordinating gene therapy research that might lead to a cure for the hexosaminidase A enzyme deficiency that causes GM2 gangliosidosis, more commonly called Tay-Sachs Disease. Great strides have been made in the scientific understanding of this disease and related gene therapies, but finding a cure will still require extensive scientific research. The New Hope Research Foundation supports this advanced research and has a mission and charter to translate this knowledge into clinical studies and to make effective therapy available to patients.
Our programs
What are the organization's current programs, how do they measure success, and who do the programs serve?
Gene Therapy Medical Research
The New Hope Research Foundation is conducting medical research in the hope of finding a genetic cure for lysosomal storage diseases that affect the central nervous system. The research is being conducted at a number of academic and hospital associated research institutes in the United States and Canada.
Where we work
External reviews

Our Sustainable Development Goals
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Goals & Strategy
Learn about the organization's key goals, strategies, capabilities, and progress.
Charting impact
Four powerful questions that require reflection about what really matters - results.
What is the organization aiming to accomplish?
The New Hope Research Foundation has been established to
coordinate, conduct, and financially support:
Research on gene therapy and the means for infusing or delivering gene vectors to
correct lysosomal enzyme deficiencies of the central nervous system,
Clinical studies intended to show safety and efficacy of gene therapy for the central
nervous system aspects of lysosomal storage diseases,
Regulatory applications required to initiate investigations of clinical studies and the
regulatory submissions required to allow patient access to proven therapies, and
Education and communication for patients and families on the clinical manifestations
and molecular basis of gangliosidosis and related diseases, on the mechanism of gene
therapy, and on the status of related research and clinical studies.
What are the organization's key strategies for making this happen?
The New Hope Research Foundation is conducting medical research to find a genetic cure for lysosomal storage diseases that affect the central nervous system. The medical research is being conducted by world experts at academic and hospital research centers. The New Hope Research Foundation serves to identify, fund, and coordinate these research activities.
What are the organization's capabilities for doing this?
The New Hope Research Foundation is being led by individuals with both a keen understanding of the diseases under investigation and a history of successful research and development projects. The New Hope Research Foundation has identified, funded, and coordinated research activities at world leading centers for genetic engineering, biochemistry, and pre-clinical studies.
What have they accomplished so far and what's next?
Medical research conducted by the New Hope Research Foundation has supported major advancements in gene therapy. Recent scientific publications have summarized some of these advancements:
Tropak, M. B. et al. Construction of a hybrid beta-hexosaminidase subunit capable of forming stable homodimers that hydrolyze GM2 ganglioside in vivo. Molecular therapy. Methods & clinical development 3, 15057, doi:10.1038/mtm.2015.57 (2016).
Karumuthil-Melethil, S. et al. Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Aav for the Treatment of Tay-Sachs Disease. Human gene therapy, doi:10.1089/hum.2016.013 (2016).
Osmon, K. J. et al. Systemic Gene Transfer of a Hexosaminidase Variant Using a scAAV9.47 Vector Corrects GM2 Gangliosidosis in Sandhoff Mice. Human gene therapy, doi:10.1089/hum.2016.015 (2016).
Keimel, J. G. et al., Gene Transfer to the Central Nervous System for Treatment of GM2 Gangliosidosis, Institute for Engineering in Medicine Annual Meeting (2015)
Sinici, I. et al. In cellulo examination of a beta-alpha hybrid construct of beta-hexosaminidase A subunits, reported to interact with the GM2 activator protein and hydrolyze GM2 ganglioside. PLoS ONE 8, e57908, doi:10.1371/journal.pone.0057908 (2013).
Clarke, J. T. et al., 2011, An open-label Phase I/II clinical trial of pyrimethamine for the treatment of patients affected with chronic GM2 gangliosidosis (Tay-Sachs or Sandhoff variants). Molecular genetics and metabolism 102, 6-12
Financials
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Operations
The people, governance practices, and partners that make the organization tick.
Connect with nonprofit leaders
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- Analyze a variety of pre-calculated financial metrics
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Connect with nonprofit leaders
SubscribeBuild relationships with key people who manage and lead nonprofit organizations with GuideStar Pro. Try a low commitment monthly plan today.
- Analyze a variety of pre-calculated financial metrics
- Access beautifully interactive analysis and comparison tools
- Compare nonprofit financials to similar organizations
Want to see how you can enhance your nonprofit research and unlock more insights? Learn More about GuideStar Pro.
NEW HOPE RESEARCH FOUNDATION
Board of directorsas of 1/12/2022
Jack Keimel
New Hope Research Foundation
Term: 2006 - 2017
Ellen Kraft
New Hope Research Foundation
Term: 2006 - 2017
Raymond Schreyer
New Hope Research Foundation
Board leadership practices
GuideStar worked with BoardSource, the national leader in nonprofit board leadership and governance, to create this section.
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Board orientation and education
Does the board conduct a formal orientation for new board members and require all board members to sign a written agreement regarding their roles, responsibilities, and expectations? Yes -
CEO oversight
Has the board conducted a formal, written assessment of the chief executive within the past year ? No -
Ethics and transparency
Have the board and senior staff reviewed the conflict-of-interest policy and completed and signed disclosure statements in the past year? Yes -
Board composition
Does the board ensure an inclusive board member recruitment process that results in diversity of thought and leadership? Yes -
Board performance
Has the board conducted a formal, written self-assessment of its performance within the past three years? No
Organizational demographics
Who works and leads organizations that serve our diverse communities? GuideStar partnered on this section with CHANGE Philanthropy and Equity in the Center.
Leadership
The organization's leader identifies as:
The organization's co-leader identifies as:
Race & ethnicity
Gender identity
Sexual orientation
No data
Disability
Equity strategies
Last updated: 12/23/2020GuideStar partnered with Equity in the Center - an organization that works to shift mindsets, practices, and systems to increase racial equity - to create this section. Learn more
- We have long-term strategic plans and measurable goals for creating a culture such that one’s race identity has no influence on how they fare within the organization.
- We engage everyone, from the board to staff levels of the organization, in race equity work and ensure that individuals understand their roles in creating culture such that one’s race identity has no influence on how they fare within the organization.